Catalent’s Elpida Therapeutics deal shows why AAV manufacturing is still a strategic choke point
Ultra-rare gene therapies need more than science. Elpida’s Catalent deal tests whether AAV manufacturing can keep patients within reach.
GenSight Biologics gets green light for GS010 in Israel: What this means for LHON patients and regulators
GenSight Biologics S.A. has received approval from Israel’s Ministry of Health to provide early access treatment with its investigational gene therapy GS010/LUMEVOQ (lenadogene nolparvovec) for individual patients diagnosed with ND4-related Leber Hereditary Optic Neuropathy (LHON). This authorization permits bilateral intravitreal injections under compassionate use protocols, with treatments expected to begin in the first quarter of […]